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Safety of a New Type of Treatment Called Gene Transfer for the Treatment of Severe Hemophilia B

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ClinicalTrials.gov Identifier: NCT00076557
Recruitment Status : Terminated
First Posted : January 28, 2004
Last Update Posted : April 4, 2007
Information provided by:

Study Description
Brief Summary:
In this study a modified virus called adeno-associated virus (AAV) will be used to transfer a normal gene for human clotting factor IX into patients with severe hemophilia B (AAV human Factor IX vector). Gene therapy is a very new medical technique being used in a number of clinical studies for diseases such as cancer and cystic fibrosis. At this time, the U.S. Food and Drug Administration has approved no gene transfer products for commercial use. To date, 8 subjects have received AAV vector in the muscle for a hemophilia B trial by intramuscular injection, and, to date, 6 subjects have been treated with AAV vector in the current hemophilia B liver trial. Eleven cystic fibrosis subjects have received AAV vector into their nasal sinuses or lungs to date. In this study, AAV human Factor IX vector will be injected into the liver using a catheter inserted into a large blood vessel (called the proper hepatic artery or the right hepatic artery).

Condition or disease Intervention/treatment Phase
Hemophilia B Genetic: Adeno-Associated Viral with Human Factor IX Phase 1 Phase 2

Study Design

Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 15 participants
Allocation: Non-Randomized
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: A Phase I Safety Study in Subjects With Severe Hemophilia B (Factor IX Deficiency) Using Adeno-Associated Viral Vector to Deliver the Gene for Human Factor IX Into the Liver
Study Start Date : January 2004

Arms and Interventions

Outcome Measures

Eligibility Criteria

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Senior)
Sexes Eligible for Study:   Male
Accepts Healthy Volunteers:   No
  • Males with severe hemophilia B with Factor IX activity level < 1% of normal.
  • Life expectancy of > 1 year.
  • Age > 18 years old.
  • Ability to give informed consent.
  • Greater than twenty exposure days of treatment with Factor IX protein.
  • No history or presence of an inhibitor to Factor IX protein.
  • Subjects must be able to receive Factor IX protein on a home infusion protocol.
  • Subjects must have a normal protime (PT).
  • Hepatitis C infected subjects will be evaluated for liver fibrosis based on liver biopsy data graded on a scale of 0–4 (Poynard et. al., 1997). Subjects who are Hepatitis C antibody and RNA positive and have not had a liver biopsy within the last 36 months will be required to have one.
  • Subjects must have low AAV titer.
Contacts and Locations

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT00076557

United States, California
Stanford University
Palo Alto, California, United States, 94305
United States, Pennsylvania
The Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
The Hemophilia Center of Western Pennsylvania
Pittsburgh, Pennsylvania, United States, 15213
Sponsors and Collaborators
Stanford University
Children's Hospital of Philadelphia
The Hemophilia Center of Western Pennsylvania
University of Washington
The University of Texas Health Science Center, Houston
University of Campinas, Brazil
Christian Medical College, Vellore, India
Royal Prince Alfred Hospital, Sydney, Australia
More Information

ClinicalTrials.gov Identifier: NCT00076557     History of Changes
Other Study ID Numbers: BB IND 9398
First Posted: January 28, 2004    Key Record Dates
Last Update Posted: April 4, 2007
Last Verified: January 2004

Keywords provided by Avigen:
Hemophilia B
Factor IX
Gene Transfer
Adeno-Associated Virus (AAV)

Additional relevant MeSH terms:
Hemophilia A
Hemophilia B
Blood Coagulation Disorders, Inherited
Blood Coagulation Disorders
Hematologic Diseases
Coagulation Protein Disorders
Hemorrhagic Disorders
Genetic Diseases, Inborn
Genetic Diseases, X-Linked